FDA, NIH Chiefs to Congress: More Support, Less Meddling

Hamburg rebuts charges that her agency hinders medical innovation.

Heads of the FDA and National Institutes of Health told a Senate panel that the public would be best served if Congress would simply give their agencies the support they need to do their jobs, with less heckling from the sidelines.

"This is a train that's on the track to the station and it's going to get somewhere, said Chairman Lamar Alexander (R-Tenn.), noting that between President Obama's Precision Medicine Initiative and the House of Representatives' 21st Century Cures legislation, Congress appears ready to act on these issues.

"I believe every single member of this committee is interested in identifying what we can do to make it easier to move those drugs, treatments, and devices from discovery all the way through to the medicine cabinet," he said.

Alexander asked Hamburg and Collins how Congress could help their agencies advance this goal.

Collins gave Alexander two concrete suggestions. First, he asked that restrictions be loosened on his employees’ ability to travel to scientific meetings. “We’re currently spending about $16 million and using hundreds of employees to go through a [paperwork] process which … has very little if any added value,” he said, noting that the process was triggered by controversy over meeting expenses at another agency. “Scientists going to conferences is a critical part of how new ideas emerge, and it’s being very much being inhibited by this heavy-handed oversight.”

A second idea, Collins continued, would be to let NIH carry funds over from one year to the next, as is permitted for the National Science Foundation and the Department of Energy. “We come up to the end of September oftentimes with money that we need to spend or it goes away. If we had the opportunity to carry that over, it wouldn’t cost another dime, and we could be more flexible in how we spend the taxpayers’ money.”

"Last year we approved the most new drugs in almost 20 years" -- 51 -- "and more orphan drugs then ever before," she said.

Contrary to common perception, her agency is not the primary reason Americans aren't receiving badly needed treatments, she said, and the U.S. is not trailing behind other countries in approvals.

Over three-quarters of new drugs brought to market in 2013 and 2014 were approved by the FDA before they were approved in the European Union, Switzerland, Canada, or Australia, she said, citing a report from the British-based Centre for Innovation in Regulatory Science.

Hamburg also noted that the FDA's process has become more flexible. Roughly 45% of new drugs were approved based on surrogate endpoints and nearly 66% followed an expedited review, she said.

"We do not want solutions based on inaccurate diagnoses," Hamburg said, referring to a frequently repeated idea that the FDA needs "an overhaul." She warned Congress against changes that would lower the agency's safety and efficacy standards for drug approvals.

"Remember that the great leaps forward in evidence-based medicine of the last 50 years have come in part because of the high standards for product approvals that Congress put in place after a series of disasters involving unsafe and ineffective medical products," she said. The reason Americans have confidence in the drugs they buy is precisely because of the agency's high standards, she added.

Hamburg argued that innovation is lagging because of a dearth of basic scientific knowledge around disease problems.

"The biggest obstacle is that scientists do not sufficiently understand the causes of Alzheimer's and other diseases to identify drug targets or identify which patients will benefit from certain drugs," she said.

The solution, suggested Hamburg, is robust public-private partnerships "bringing the best minds together to develop the science that we need."

Collins echoed Hamburg's position. He said that the most common reason for failure in drug development is that scientists choose the wrong pathways to target. Last year's launch of the Accelerating Medicines Partnership (AMP), a collaboration of the FDA, NIH, industry and nonprofits is focused on changing that, he said.

The "pre-competitive" public-private partnership focuses on three disease areas: Alzheimer's, type 2 diabetes, and autoimmune diseases -- such as rheumatoid arthritis and lupus. The scientists work together to decide the best targets for therapeutic intervention.

In addition to touting new initiatives like AMP and Precision Medicine, Collins spoke about the repercussions of last year's sequestration. He said the threat of more budget cuts "hangs over us like a dark cloud."

The NIH has lost 23% of its purchasing power since 2003, Collins said, forcing the NIH to decline about half of the proposed studies that historically it would have funded.

Sen. Elizabeth Warren (D-Mass.) agreed with Collins and Hamburg. She said that while certain changes could improve the speed of drug approvals -- such as eliminating some of the bureaucracy -- lowering regulatory standards shouldn't be one of them.

"We could abolish the FDA tomorrow, and we'd see tons of new products on the market," said Warren, but Congress' goal isn't to inflate industry profits and risk patients' safety. "We don't want another Vioxx."

Warren said that instead of attacking the FDA's processes, Congress should focus on the NIH and pay the organization more than lip service.

"Congress has decimated the NIH's budget, single-handedly choking off support for projects that could lead to the next major breakthrough in Alzheimer's and many other diseases," Warren said.

Dismantling the FDA won't produce new cures, she added.

"If we're serious about better health for children and seniors then Congress has to step up and make a real commitment of real dollars for scientific research."

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